METHODS: Relevant articles were identified through searches of MEDLINE (1966-June 2005) and International
Pharmaceutical Abstracts (1970-June 2005). The search terms included
pegaptanib sodium,
Macugen,
age-related macular degeneration, and
choroidal neovascularization. The reference lists of identified articles were reviewed for additional publications, and further information was obtained from the manufacturer of
pegaptanib. Included studies were review articles and Phase II, III, and IV clinical trials, with preference given to available Phase III studies.
RESULTS: Only 1 research group has evaluated the tolerability and efficacy of
pegaptanib in patients with neovascular ARMD. The
VEGF Inhibition Study in Ocular Neovascularization involved 2 concurrent randomized trials of intravitreous
injections of
pegaptanib 0.3 mg (n = 294), 1 mg (n = 300), and 3 mg (n = 296) compared with
sham injections (n = 296) every 6 weeks for 54 weeks in patients with neovascular ARMD. Assessments were conducted at 6, 12, 18, 24, 30, 42, 48, and 54 weeks. The primary end point was the proportion of patients losing <15 letters on the study eye chart at 54 weeks. This end point was achieved in 70%, 71%, and 65% of patients who received
pegaptanib 0.3 (P < 0.001), 1 (P < 0.001), and 3 mg (P = 0.03), respectively, compared with 55% of those receiving the
sham injections. Significant improvements in visual acuity with
pegaptanib compared with the
sham-injection group were seen at all time points (0.3 and 1 mg: P < 0.002; 3 mg: P < 0.05). The
sham-injection group was twice as likely to have severe vision loss (loss of > or =30 letters or 6 lines on the eye chart) compared with those receiving
pegaptanib 0.3 or 1 mg (P < 0.001). Adverse events reported significantly more often in the
pegaptanib group compared with the
sham-injection group included
vitreous floaters (33% vs 28%, respectively; P < 0.001), vitreous opacities (18% vs 10%; P < 0.001), and anterior-chamber
inflammation (14% vs 6%; P = 0.001). Injection-related adverse events during the first year of
pegaptanib treatment included
endophthalmitis in 12 (1.3%) patients,
retinal detachment in 6 (0.7%) patients, and traumatic injury to the lens in 5 (0.6%) patients.
CONCLUSIONS: There are few published clinical data on
pegaptanib. In 2 clinical comparisons with
sham injections,
pegaptanib was well tolerated and effective in slowing the decline in visual acuity in patients with neovascular ARMD. This agent may be considered an option for the treatment of neovascular ARMD.